When most people think of superheroes they envision
larger than life characters cloaked in masks and capes. But to
the families of children with Phenylketonuria (PKU) -- a rare
and debilitating metabolic disorder -- their hero has salt
& pepper hair and wears a lab coat.

His name is
Ray Stevens and he'll humbly tell you that he's just a
researcher at Scripps. But that hardly sums up the man who is
leading the way to improving the lives of thousands of
children.

This holiday season,
continue to help all the heroes at Scripps
Research provide
hope.

Because of friends like you,
a second PKU drug developed by Ray Stevens will go
into clinical trials next year… eventually giving
doctors more treatment options.

It's just
one more piece of hope that you helped Ray give
these families.

It all began in the 1990's when Ray was conducting
basic metabolic research at Scripps Research and discovered
mutations in the gene that is responsible for PKU. At that
point, the only way to control PKU was a strict, low-protein
diet. Because PKU patients are unable to process a part of a
protein called phenylalanine (Phe), which is found in almost
all foods, their lives become drastically limited. However, if
Phe levels get too high, it can cause severe mental
retardation.

But Ray's discovery finally offered a promising
opportunity to find a treatment and maybe even a cure. The
only problem was funding. Because PKU affects less than
200,000 people a year, it's classified as an "orphan" disease.
And these diseases typically don't get "adopted" by the
pharmaceutical industry because they provide little financial
incentive.

For families dealing with PKU, and all of us at The
Scripps Research Institute, this was unacceptable. But without
funding, the opportunity to develop a new drug seemed
unlikely.

And then fate brought the scientist and
these families together. It started with an invitation to
discuss his PKU research at a fundraiser. Soon, Ray's
determination to find a treatment turned into a passion. When
he wasn't in his Scripps Research lab, he was participating in
walk-a-thons, PKU picnics, and an array of fundraising events…
all to raise money for his life-changing research.

The hope that Ray's research offered these families
fueled a truly amazing outcome. From that initial fundraising
effort, Scripps Research was able to collect enough data to
apply for, and receive, a 1.2 million dollar grant from the
National Institutes of Health to develop a drug for PKU! And
it's most fitting that Kuvan -- the first-ever drug
for PKU -- will finally be available to patients this month.

It's an incredibly wonderful holiday
miracle for thousands of children.